On the heels of a unanimous FDA panel vote last month in favor of its application, Epizyme’s rare cancer med Tazverik scored U.S. approval Thursday.
Tazverik won approval to treat patients 16 and older with epithelioid sarcoma, a rare soft tissue cancer that often occurs in young adults. Patients with cancer have previously had no targeted treatment options, so treatment was based around surgical removal of their tumors and chemotherapy. The drug is only approved for patients whose cancer can't be completely removed by surgery.
Epithelioid sarcoma typically starts in soft tissues under an extremity, the FDA says, but it can also start elsewhere in the body. Epizyme estimates about 800 patients suffer from the disease in the U.S., and that about 300 patients will be eligible for the treatment. Given the small patient population, SVB Leerink analysts Andrew Berens predicted some investors "may be disappointed" by the drug's $15,500-per-month list price, which is lower than its peers have gone.
"Some investors ... had assumed the company could price at a premium level in this ultra-rare disease, similar to Blueprint Medicines' recent approval for Ayvakit ($32K per month WAC)," he wrote in a Monday research note.
But Epizyme selected its price with an eye toward the future, Jefferies analyst Michael Yee noted in his own dispatch to clients. The company is aiming for an approval in follicular lymphoma and submitted that application to the FDA last month. Epizyme priced its med for the U.S. follicular lymphoma market, which numbers more than 10,000 patients, rather than the "ultra-orphan" indication where it scored its initial approval, Yee pointed out. The company further plans to explore the drug's potential in other uses.
For now, launch preparations are underway and the company expects the drug to be available within 10 business days, Epizyme chief strategy and business officer Matthew Ros said on a conference call Thursday. The company has a "nimble" field-based team of 19 oncology experts, including business directors, medical science liaisons, market access personnel and others, he said.
The FDA nod follows a unanimous vote in favor of the drug at an FDA advisory committee meeting last month. The panel discussed the med’s relatively low overall response rate of 15%, but panelists noted that the number wasn’t the only factor to consider in the review. Several highlighted long responses—67% of patients who responded saw benefits that lasted more than six months—and said the drug could offer meaningful benefit for patients fighting an aggressive cancer with no other options.
Before the panel vote, FDA reviewers raised some of their own doubts about the drug, highlighting worries over efficacy, safety and the size of the phase 2 trial Epizyme ran in order to submit the drug.